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Updated: Oct 25, 2025
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Isolation and Transplantation of Different Aged Murine Thymic Grafts.
Published on: May 13, 2015
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Experience with cultured thymus tissue in 105 children
M Louise Markert1, Stephanie E Gupton2, Elizabeth A McCarthy2
1Department of Pediatrics, Division of Allergy, Immunology, and Pulmonary Medicine, Duke School of Medicine, Durham, NC; Department of Immunology, Duke School of Medicine, Durham, NC.
The Journal of Allergy and Clinical Immunology
|August 7, 2021
Summary
Cultured thymus tissue (CTT) offers a promising treatment for congenital athymia, a severe immune deficiency. This therapy facilitates T-cell development, improving survival rates and reducing infections in affected patients.
Area of Science:
- Immunology
- Developmental Biology
- Genetics
Background:
- Congenital athymia is a severe primary immunodeficiency with no approved therapies, leading to high early mortality.
- Associated conditions include DiGeorge syndrome, CHARGE syndrome, and FOXN1 deficiency.
- Immune dysregulation and susceptibility to infection characterize this condition.
Purpose of the Study:
- To evaluate the efficacy of cultured thymus tissue (CTT) in treating patients with congenital athymia.
- To assess T-cell development, survival rates, and adverse events in treatment-naive patients receiving CTT.
Main Methods:
- Analysis of data from 10 prospective, single-arm, open-label studies (1993-2020).
- Inclusion of 95 treatment-naive patients with congenital athymia in the efficacy analysis set.
- Monitoring of T-cell development, infections, and adverse events post-CTT administration.
Main Results:
- A 1-year survival rate of 77% was observed in patients receiving CTT.
- Immune reconstitution, evidenced by naive T-cell development, occurred typically within 6-12 months.
- Rates of infections and autoimmune complications decreased significantly after one year.
Conclusions:
- Cultured thymus tissue (CTT) treatment leads to the development of naive T cells and improved survival in congenital athymia.
- The therapy demonstrates potential for immune reconstitution, mitigating life-threatening infections.
- CTT represents a viable therapeutic strategy for congenital athymia, enhancing long-term patient outcomes.
